Megan Dorrell, PharmD, BCACP’s Post

Our Phase 2a HuMAIN trial met the primary endpoint of weight reduction in patients w/ obesity-related heart failure. We're sharing detailed results highlighting our investigational therapy's potential to be the first disease-modifying #HFpEF treatment by enabling fat-specific weight loss while preserving muscle at this year's #HFSA Annual Scientific Meeting. Read more: https://bit.ly/4fArk2O

Soligenix reports positive results from phase 2a study of SGX302 in patients with mild-to-moderate psoriasis https://lnkd.in/dBxBwa-5

New Weight Loss Pill Surpasses Semaglutide in Clinical Trial

I recently spoke with NBC News about Novo Nordisk’s experimental weight loss pill, #amycretin, which showed promising results in a Phase 1 trial. Participants lost up to 13% of their body weight in just three months—double the rate of drugs like Ozempic and Wegovy. Amycretin mimics two hormones to reduce appetite and promote fullness, with the added benefit of amylin. While these early results are impressive, more research is needed to assess long-term effects and side effects. It’s crucial to consider not just how much weight is lost, but how it’s lost. Sustainable, healthy weight loss is key to maintaining results and preserving lean mass. “Did patients lose weight more rapidly because they were less hungry or because they were sick?” Although amycretin shows potential, it hasn’t been directly compared to existing drugs, and the findings haven’t been published in a peer-reviewed journal. https://lnkd.in/e7JfNDkz

🚀 Exciting News Alert! 🚀 Beckley Psytech reports positive initial findings from their Phase IIa clinical study (NCT05660642) of BPL-003 (5-MeO-DMT) for Treatment Resistant Depression (TRD). BPL-003 is benzoate salt formulation of 5-MeO-DMT (Mebufotenin), administered intranasally. In this open-label study, a single 10mg dose of BPL-003 was well-tolerated and demonstrated rapid, durable efficacy in patients with TRD. Key highlights include: ·      Rapid antidepressant response observed in 55% of patients the day after dosing. ·      55% of patients in remission from depression at day 29 and 45% at day 85 post-dosing. “Acute effects were resolved and patients were deemed dischargeable within an average time of less than 2 hours. This signals the potential for a shorter treatment duration and reduced resource burden for healthcare systems compared to other psychedelic treatments currently in development.” #psychedelics #clinicaltrials #mentalhealth #treatmentresistentdepression

Jayme Locke and a team of researchers at the University of Alabama at Birmingham have discovered that immunosuppressant’s that are FDA approved—and that are currently in use for human-to-human transplants—are the optimal choices for an immunosuppression regime for pig-to-human kidney transplants. To learn more about their research and what their hopes are for the future of xenotransplantation, read the full article below! #xenotransplantation #kidneytransplant

#BreakingNews: In one of the most highly anticipated #clinicaltrial readouts this year, an #RNAsilencing treatment developed by Alnylam Pharmaceuticals reduced the risk of death, heart-related hospital visits and hospitalizations by 28% compared to placebo in patients with a #heartmuscledisease. The results, announced Monday, pave the way for #Alnylam’s therapy #vutrisiran to become the first #RNAsilencingtherapy on the market for #ATTRamyloidosis with #cardiomyopathy, a condition in which misfolded proteins build up in the heart and can result in heart failure. If approved, Alnylam could enter a multibillion-dollar market currently dominated by Pfizer. Vutrisiran is already approved as Amvuttra for ATTR amyloidosis with polyneuropathy, where the misfolded proteins affect the nerves. Alnylam believes that it can potentially reach 10 times more patients in the expanded indication, as the heart manifestation of the disease has been estimated to impact as many as 500,000 globally. “We believe with the data we have in hand, this will become the new standard of care in patients with ATTR cardiomyopathy,” Alnylam CEO Dr. Yvonne Greenstreet told Endpoints News on Saturday. “This is really, really tremendous data and clearly a huge opportunity for Alnylam.” Read more from Endpoints News 👇🏼

Today, AbbVie announced positive top-line results from their Phase 3 study, SELECT-GCA. Upadacitinib (RINVOQ, 15 mg, once daily), paired with a 26-week steroid taper regimen, achieved its primary endpoint of sustained remission from week 12 through week 52 in adults with giant cell arteritis (GCA) with the safety profile remaining consistent with that in approved indications. Key secondary endpoints were also met, with a larger percentage of patients receiving Upadacitinib with the 26-week steroid taper regiment achieving sustained complete remission compared to placebo, and a lower percentage of patients experienced at least one disease flare through week 52 than placebo. However, the study showed that Upadacitinib 7.5 mg did not meet the primary or any of the secondary endpoints. Read more: https://lnkd.in/ejmhxSRF   #GiantCellArteritis #GCA #Rheumatology #AbbVie

https://lnkd.in/e7HF4EqW We are a MDA center of excellence, recognized for both muscle dystrophy and ALS. #als is cruel. There is no question. The very human desire for hope is real. These are very tough moral and medical conversations. In the end: the data has to tell the story. We are obliged to our patients to be inventive but also rigorous. #neuromuscular #drugdevelopment #medicine

Synergy Healthcare is thrilled to share groundbreaking developments in the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The Food and Drug Administration (FDA) is on the verge of approving the first-ever medicine specifically designed to combat this serious liver disease. Developed by Madrigal Pharmaceuticals, the pill, known as resmetirom, targets a protein in the liver, effectively reducing liver fat, inflammation, and scarring associated with MASH. If approved, resmetirom will mark a significant milestone in healthcare, offering hope to patients with advanced stages of MASH where current treatments have shown limited effectiveness. At Synergy Healthcare, we pride ourselves on being at the forefront of clinical research, especially in metabolic and neurological diseases. The potential approval of resmetirom represents a significant step forward in addressing the unmet medical needs of patients suffering from MASH. The addressable market for drugs to treat advanced MASH is substantial, with an estimated 315,000 individuals in need of effective treatment. Resmetirom's innovative approach and promising clinical trial results position it as a potential game-changer in the field of liver disease treatment. We commend the dedication and perseverance of researchers and scientists, including those at Madrigal Pharmaceuticals, for their tireless efforts in bringing new therapeutic options to patients in need. Stay tuned for more updates as we eagerly await the FDA's decision on resmetirom, and let's continue our collective efforts to advance healthcare and improve patient outcomes! 💊💪 #SynergyHealthcare #ClinicalResearch #MASHTreatment #Resmetirom #HealthcareInnovation #LiverDiseaseTreatment #FDAApproval #MedicalBreakthroughs 🔗 https://lnkd.in/ekjzn4uy